RZ-004

RZ-004 is an investigational gene therapy developed for the treatment of autosomal dominant retinitis pigmentosa caused by mutations in the rhodopsin (RHO) gene. It utilizes an adeno-associated virus (AAV) vector to deliver a trans-splicing ribozyme specifically targeting mutant rhodopsin mRNA. The ribozyme edits and reprograms the mutant mRNA into its normal form, aiming to restore normal protein function and address various mutations within each patient using a single therapeutic strategy. This approach offers potential as a disease-modifying treatment for patients with this genetic disorder[1][2][3][4][7].

Other names

RHO gene therapy